Jacques P. Tremblay received a B.Sc. in Biochemistry from McGill University in 1970, and a Ph.D. in Neuroscience from UCSD (University of California in San Diego) in 1974. From 1975 to 1976, he was a postdoctoral fellow at the Laboratory of Neurobiology of l’Hôpital de l’Enfant-Jésus. Subsequently, he spent his entire career at Laval University: Professor under grant from 1976 to 1981 in the Department of Anatomy; Assistant Professor from 1981 to 1985; full Professor from 1985; Director of the Department of Anatomy from 1987 to 1997, and Professor of the Department of Molecular Medicine from 2010 to now. He is currently a regular researcher at the Axis of Neuroscience of the CHU Research Center of Quebec-Université Laval.

Development of a treatment for Duchenne Muscular Dystrophy (DMD)

DMD is due to a mutation of the gene coding for the dystrophin protein. This mutation leads to an absence of this protein under the membrane of muscle fibers. His laboratory is renowned for his work on the transplantation of normal allogeneic myoblasts as a treatment for DMD. His Phase I clinical trial for this therapy showed that this transplant restores the expression of this protein in the patient’s muscle fibers. In 2006, Dr. Tremblay received the Henry Friesen Award from the Royal College of Physicians and Surgeons of Canada for his work on this therapy. His group is currently conducting a Phase I / II clinical trial on this therapy. In addition, his group is currently using CRISPR / Cas9 technology to correct the dystrophin gene, creating an additional deletion to produce a hybrid exon of the dystrophy gene, which not only restores the expression of dystrophin but also produces dystrophin with a normal structure.

Development of a treatment for Friedreich’s Ataxia

Dr. Tremblay’s group has also been conducting research on Friedreich’s Ataxia since 2010. This disease is due to an elongation of the GAA trinucleotide repeat in intron 1 of the frataxin gene, which reduces expression of this protein, leading to the death of neurons and cardiomyocytes that induce neurological and cardiac symptoms. His group demonstrated that the expression of frataxin is increased by targeting the promoter of this gene with TALE-VP64 proteins. In addition, it has also demonstrated that it is possible to suppress trinucleotide repetition by cutting with the CRISPR / Cas9 system before and after this repeat.

Development of a treatment for Alzheimer’s disease

This group also uses CRISPR / Cas9 technology to develop a treatment for Alzheimer’s disease. This disease is due to the abnormal metabolism of the APP protein (Amyloid Precursor Protein) which leads to the formation of beta-amyloid peptides that form plaques. The formation of these peptides can be greatly reduced by the A673T mutation of the APP gene observed in a small portion of Iceland’s population. Dr. Tremblay’s group has demonstrated that this mutation could be produced with the CRISPR / Cas9 system.

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271 entries « 2 of 28 »

Ouellet DL, Cherif K, Rousseau J, Tremblay JP

Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia.

Journal Article

Gene Ther, 24 (5), pp. 265-274, 2017, ISSN: 0969-7128.

Abstract | Links:

Ouellet DL, Duchêne B, Iyombe-Engembe JP, Tremblay JP

Advances & challenges of using CRISPR-Cas9 gene editing for treating Duchenne muscular dystrophy

Journal Article

Cell Gene Ther Insights, 3 (1), pp. 53-58, 2017, ISSN: 2397-0545.

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Skuk D, Tremblay JP

CD56+ Muscle Derived Cells but Not Retinal NG2+ Perivascular Cells of Nonhuman Primates are Myogenic after Intramuscular Transplantation in Immunodeficient Mice

Journal Article

J Stem Cell Res Ther, 7 (2), 2017.

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Iyombe-Engembe JP, Tremblay JP

The advances and challenges of Gene Therapy for Duchenne Muscular Dystrophy

Journal Article

J Genet Med Gene Ther, 1 , pp. 19-36, 2017.

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Skuk D, Tremblay JP

The Process of Engraftment of Myogenic Cells in Skeletal Muscles of Primates: Understanding Clinical Observations and Setting Directions in Cell Transplantation Research.

Journal Article

Cell Transplant, 26 (11), pp. 1763-1779, 2017, ISSN: 0963-6897.

Abstract | Links:

Iyombe-Engembe JP, Ouellet DL, Barbeau X, Rousseau J, Chapdelaine P, Lague P, Tremblay JP

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method.

Journal Article

Mol Ther Nucleic Acids, 5 , pp. e283, 2016.

Abstract | Links:

Chapdelaine P, Gerard C, Sanchez N, Cherif K, Rousseau J, Ouellet DL, Jauvin D, Tremblay JP

Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice.

Journal Article

Gene Ther, 23 (7), pp. 606-14, 2016, ISSN: 0969-7128.

Abstract | Links:

Skuk D, Tremblay JP

Confirmation of donor-derived dystrophin in a duchenne muscular dystrophy patient allotransplanted with normal myoblasts.

Journal Article

Muscle Nerve, 54 (5), pp. 979-981, 2016, ISSN: 0148-639X.

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Sanchez N, Chapdelaine P, Rousseau J, Raymond F, Corbeil J, Tremblay JP

Characterization of frataxin gene network in Friedreich's ataxia fibroblasts using the RNA-Seq technique.

Journal Article

Mitochondrion, 30 , pp. 59-66, 2016, ISSN: 1567-7249.

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Tremblay JP, Iyombe-Engembe JP, Duchene B, Ouellet DL

Gene Editing for Duchenne Muscular Dystrophy Using the CRISPR/Cas9 Technology: The Importance of Fine-tuning the Approach.

Journal Article

Mol Ther, 24 (11), pp. 1888-1889, 2016, ISSN: 1525-0016.

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271 entries « 2 of 28 »
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Active projects

  • Centre de recherche du CHU de Québec - Université Laval, Subvention, Centre hospitalier universitaire de Québec - Université Laval, Centres de recherche affiliés, from 2017-01-01 to 2099-12-31
  • Centre thématique de recherche en neurosciences, Subvention, Institutionnel - BDR, BDR - Centres de recherche reconnus, from 1999-06-01 to 2023-05-01
  • Correction by CRISPR base editing of point mutations responsible for Duchenne Muscular Dystrophy, Subvention, The Foundation for gene and cell therapy Jesse's Journey, from 2020-06-01 to 2022-05-31
  • Deciphering the role of DCIR in HIV-1 pathogenesis, Subvention, Instituts de recherche en santé du Canada, Subvention Projet, from 2018-04-01 to 2023-03-31
  • Development of a treatment of Alzheimer based on the editing of the Amyloid Precuror Protein gene with the CRISPR system., Subvention, Weston Brain institute, Transformational Research, from 2016-01-15 to 2020-09-30
  • Phase I/II clinical trial of myoblast transplantation to Duchenne Muscular Dystrophy patients., Subvention, Instituts de recherche en santé du Canada, Subvention de fonctionnement, from 2013-10-01 to 2022-03-31
  • Removal of the GAA repeat with the CRISPR/Cas9 system in Friedreich patient cells and in the YG8sR mouse model, Subvention, Instituts de recherche en santé du Canada, Subvention Projet, from 2019-10-01 to 2024-09-30
  • Using extracellular vesicles to deliver therapeutic proteins for various ataxia, Subvention, National Ataxia Foundation, Research Seed Money, from 2020-03-01 to 2021-07-01

Recently finished projects

  • Acquisition d'une centrifugeuse , Subvention, Fondation du CHU de Québec, from 2019-10-28 to 2020-03-31
  • Correction of the dystrophin gene with Zinc Finger Proteins and TAL effector nuclease, Subvention, Instituts de recherche en santé du Canada, Subvention de fonctionnement, from 2012-10-01 to 2020-03-31
  • Étude sur des souris modèles de l'Ataxie de Friedreich d'une thérapie basée sur l'induction de l'expression du gène de la fraxine avec des TALEs-SunTag, Subvention, Le Grand Défi Pierre Lavoie, from 2018-10-01 to 2019-09-30
  • Phase I/II clinical trial delivering myoblasts to DMD patients, Subvention, The Foundation for gene and cell therapy Jesse's Journey, from 2019-10-31 to 2020-05-01
  • Préparation d'un document de présentation sur la recherche en génétique humaine , Subvention, Fondation du CHU de Québec, from 2019-11-16 to 2020-03-31
  • Removal of the GAA repeat with the CRISPR/Cas9 system in Freidreich patient cells and in the YG8sR mouse model, Subvention, Association française contre les myopathies, from 2018-07-01 to 2020-06-30
  • Removal of the GAA repeat with the CRISPR/Cas9 system in Freidreich patient cells and in the YG8sR mouse model, Subvention, Ataxie Canada, from 2018-07-01 to 2020-06-30
  • Utilisation de la technologie CRISPR / Cas9 dans le développement d'un traitement permanent contre la maladie d'Alzheimer, Subvention, Ministère des Relations internationales et de la Francophonie, Coopération bilatérale Québec-Brésil, from 2018-04-01 to 2020-03-30
Data provided by the Université Laval research projects registery